A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001
试验速览
- 阶段
- 1 期
- 入组人数
- 28
- 试验地点
- 1
- 主要终点
- Tolerance of extended treatment
研究概览
简要总结
Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment.
详细描述
Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment. The same safety assessments as in the CMD-2016-001 study will be conducted after each cycle of treatment and the same efficacy assessments as in the CMD-2016-001 study will be conducted after every 3 cycles of treatment. Treatment will continue until the first to occur of (1) Investigator considers the patient is no longer deriving benefit from CuATSM treatment, (2) patient develops dependence on mechanical ventilation where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use, or (3) the patient withdraws consent.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent prior to initiation of any study-specific procedures and treatment
- •Documented completion of protocol-specific assessments following completion of six 28-day treatment cycles in study CMD-2016-001
- •Principal Investigator considers the patient would benefit from continued treatment with Cu(II)ATSM
- •Not taking riluzole or on the same (or lower) dose used during the CMD-2016-001 study
- •Adequate bone marrow reserve, renal and liver function
- •Women and men with partners of childbearing potential must take effective contraception while on study treatment
排除标准
- •Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g.,malabsorption) deemed to jeopardize intestinal absorption of study drug
- •Dependence on mechanical ventilation (invasive or non-invasive) for any part of day or night, where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use
- •Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures
- •Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6
研究组 & 干预措施
Cu(II)ATSM
Cu(II)ATSM administered once daily
干预措施: Cu(II)ATSM (Drug)
结局指标
主要结局
Tolerance of extended treatment
时间窗: 24 months
safety
次要结局
- Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)(24 months)
- Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score(24 months)
- Treatment-related change in respiratory function by sniff nasal pressure (SNP) test(24 months)
- Treatment-related change in respiratory function by seated forced vital capacity (FVC)(24 months)
- Treatment-related change in quality of life by ALSSQOL-R score(24 months)